Vivo-Morpholinos for adult animals

[Jon Moulton]

The availability of Vivo-Morpholinos makes the specific and effective activities of Morpholino oligos available in many traditional animal model systems where Morpholinos were not effective in the past due to delivery issues. Highly specific Morpholino translation blocking, splice blocking and miRNA maturation inhibition are now feasible in adult rodents, allowing knockdowns without the known widespread off-target gene modulation associated with RNAi technologies and RNase-H dependent antisense.

Vivo-Morpholinos enter the cells of adult organisms when administered by I.V. injection. Initial results in mouse, rat and adult zebrafish show activity of splice-blocking and translation-blocking Morpholinos in tissues after introduction of Vivo-Morpholinos into the blood. Delivery efficacy varies by tissue type, but this is far better activity than is seen with bare Morpholino oligos.

Vivo-Morpholinos are Morpholino oligos covalently linked to octaguanidinium dendrimers. The dendrimers are synthesized on the Morpholino oligo while the oligos are still attached to their synthesis resin, then capped with guanidiniums. The oligos are then cleaved from the resin and the bases deprotected. This means that existing oligos cannot be modified to be Vivo-Morpholinos; addition of a delivery moiety must be specified prior to synthesis. Vivo-Morpholinos are available on the Gene Tools online store and are described on the Gene Tools website (Vivo-Morpholinos - Gene Tools, LLC), where you can find links to some gel images from splice-modification experiments in mouse.

After many years of research we are excited to finally offer a delivery moiety that brings Morpholino techniques to life science researchers working in adult animals.